As an alternative to C5 inhibition with Eculizumab, several AP-targeted therapeutics, including the fD inhibitor Danicopan (ACH-4471) (46) and the fB inhibitor Iptacopan (LNP023) (38) are currently being evaluated in the clinic, and successful phase 2 clinical trials with both agents against PNH have been recently reported (47, 48). The gene discussed is ELP1; the disease is paroxysmal nocturnal hemoglobinuria.